AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101
Agency is supportive of proposed analytical methods and comparability protocol
AveXis anticipates initiating U.S. SMA Type 1 and Type 2 trials in Q3 2017 with product from new process
AveXis manufacturing facility is fully operational for GMP product production
Conference call and webcast today at 4:30 p.m. EDT
Chicago, Ill. (June 14, 2017) AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced alignment with the U.S. Food and Drug Administration (FDA) on the companys Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following the receipt of minutes from the Type B Chemistry Manufacturing and Controls (CMC) meeting. This alignment includes support for the proposed commercial manufacturing process, the proposed analytical methods and corresponding
qualification and validation plans inclusive of key release assays such as potency, purity and identity and the proposed comparability protocol, which helps assess how similar the product derived from the GMP process is to the original product used in the Phase 1 trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1.
Key components of the scalable, GMP commercial manufacturing process are as follows:
· AveXis will continue to utilize HEK293 cells and an adherent cell line.
· The commercial-scale GMP process will not include the use of Hyperstacks for AAV9 vector production.